Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans.

Maurizi, Antonio; Patrizii, Piergiorgio; Teti, Anna; Sutera, Flavia Maria; Baran-Rachwalska, Paulina; Burns, Chris; Nandi, Uttom; Welsh, Michael; Torabi-Pour, Nissim; Dehsorkhi, Ashkan; Saffie-Siebert, Suzanne

Maurizi, Antonio; Patrizii, Piergiorgio; Teti, Anna; Sutera, Flavia Maria; Baran-Rachwalska, Paulina; Burns, Chris; Nandi, Uttom; Welsh, Michael; Torabi-Pour, Nissim; Dehsorkhi, Ashkan; Saffie-Siebert, Suzanne.

Afiliación

Maurizi A; Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy.
Patrizii P; Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy.
Teti A; Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy.
Sutera FM; SiSaf Limited, Guildford, UK.
Baran-Rachwalska P; SiSaf Limited, Guildford, UK.
Burns C; PorousSilicon, Salzburg, Austria.
Nandi U; SiSaf Limited, Guildford, UK.
Welsh M; SiSaf Limited, Guildford, UK.
Torabi-Pour N; SiSaf Limited, Guildford, UK.
Dehsorkhi A; SiSaf Limited, Guildford, UK.
Saffie-Siebert S; SiSaf Limited, Guildford, UK.

Mol Ther Nucleic Acids ; 33: 925-937, 2023 Sep 12.

Article en En | MEDLINE | ID: mdl-37680985

Palabras clave

MT: delivery strategies; autosomal dominant osteopetrosis; bone resorption; delivery nanoparticles; genetic bone diseases; osteoclasts; silicon; therapeutic siRNA; therapy

Texto completo

Añadir a Mi BVS

Imprimir

XML

PubMed Links

Buscar en Google

Texto completo: 1 Bases de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2023 Tipo del documento: Article País de afiliación: Italia